13 September 2021 - Natural history analysis shows significant improvement in event-free survival and transplant-free survival.
Mirum Pharmaceuticals today announced the submission of a marketing authorisation application for the treatment of cholestatic liver disease in patients with Alagille syndrome to the European Medicines Agency.
In conjunction with the Alagille syndrome submission, Mirum’s marketing authorisation application for progressive familial intrahepatic cholestasis type 2 (PFIC2) was withdrawn with plans to re-submit after availability of results from the ongoing MARCH-PFIC Phase 3 study in a broader set of PFIC sub-types and with higher doses of maralixibat.